Runge D M, Runge D
Department of Pathology, University of Pittsburgh.
Fortschr Med. 1995 Aug 30;113(24):337-40.
Somatic gene therapy represents an attempt to cure the genetic defects responsible for metabolic and neurological diseases, a major application is the treatment of cancer. Phase I studies have already been implemented. Although advances have been made, a number of central questions have yet to be answered before somatic gene therapy can be applied safely and effectively in patients. The development of effective techniques in gene transfer and cell transplantation is essential for successful therapy. Since this is a new area of research and development, and since genetically inherited diseases differ widely, many strategies are currently tested in animal models. Some of these strategies are discussed in the present review.
体细胞基因治疗旨在治愈导致代谢和神经疾病的基因缺陷，其一个主要应用领域是癌症治疗。目前已经开展了一期研究。尽管取得了一些进展，但在体细胞基因治疗能够安全有效地应用于患者之前，仍有许多核心问题有待解答。基因转移和细胞移植有效技术的开发对于成功治疗至关重要。由于这是一个新的研发领域，而且遗传性疾病差异很大，目前许多策略正在动物模型中进行测试。本综述将讨论其中一些策略。
